TN-201 - (MyPeak-1)

June 3, 2026: Tenaya Therapeutics, Inc. (NASDAQ: TNYA) reported new interim safety and efficacy data from the MyPEAK-1 Phase 1b/2 trial (NCT05836259) of TN-201 gene therapy for MYBPC3-associated nonobstructive hypertrophic cardiomyopathy (HCM). Data (May 2026 cutoff) include 78–104 weeks follow-up for Cohort 1 (3E13 vg/kg; 3 patients) and 26–52 weeks for Cohort 2 (6E13 vg/kg; 4 patients). Safety across 7 dosed patients showed TN-201 was generally well tolerated with no dose-limiting toxicities and no new safety events since the prior readout; all patients have tapered off immunosuppression.

Efficacy-evaluable patients (n=6; 3 per cohort) showed improvements across multiple parameters: all six reduced left ventricular mass index (durable through two years for the first two Cohort 1 patients) and five of six reduced one or more wall-thickness measures; NYHA class improved by ≥1 in five of six (all five now Class I); KCCQ-CSS improved 12–56 points in four of six (Cohort 2 mean +36); and functional capacity improved by ≥1 of 6MWT/CPET in three patients (6MWT +50 to +255 meters; one Cohort 2 patient improved pVO2). Tenaya noted earlier/more substantial signals at the higher dose.

Regulatory catalysts: TN-201 received EMA PRIME designation; TN-201 for biallelic pediatric patients was accepted into FDA’s Rare Disease Evidence Principles (RDEP) process. Next: additional long-term follow-up data planned in 2H 2026 and a regulatory update by year-end.